Insmed Incorporated’s (INSM) shares skyrocketed 42.6% after it received breakthrough therapy designation for Arikayce from the FDA. The designation was granted to Arikayce for curing patients suffering from treatment resistant nontuberculous mycobacterial (:NTM) lung infections.
Breakthrough therapy designation is a status provided under the Food and Drug Administration Safety and Innovation Act (:FDASIA) of 2012 to potential candidates that show superiority over existing therapies for the treatment of serious or life threatening diseases or conditions. The designation helps to speed up the development and review process for experimental drugs targeting serious and life-threatening diseases. A candidate with breakthrough therapy designation could benefit from the FDA’s existing fast track development program features as well as more intensive FDA guidance on efficient development of the candidate.
Insmed now intends to discuss with the U.S. regulatory authority the future pathway of Arikayce. The FDA’s decision was based on the findings from the phase II study on Arikayce in NTM patients.
The phase II study evaluated Arikayce plus standard-of-care treatment vis-à-vis standard-of-care treatment plus placebo in treatment resistant NTM lung infected adults for 84 days. These patients were on American Thoracic Society/Infectious Disease Society of America (ATS/IDSA) guideline therapy for a minimum period of six months before screening.
Last month, Insmed announced positive additional data from the phase II study on the candidate. Data from the study revealed that 21 of the 68 patients eligible were culture negative for NTM at the end of 168 days. In addition, the number of patients with negative cultures increased from the double-blind phase of the trial.
However, in March this year, Arikayce failed to meet the primary objective of the phase II study of a pre-specified level for statistical significant difference in mycobacterial density on a seven-point scale from baseline to the end of the randomized portion of the study.
Arikayce already enjoys orphan drug designation for treating NTM both in the U.S. and EU and it also enjoys fast track designation for NTM in the U.S.
Arikayce is the lead candidate at Insmed which currently has no approved drug in its portfolio. Hence, the successful development of the candidate is crucial for the company’s growth. We expect investor focus to remain on further updates on Arikayce.
Insmed currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the pharma sector include Regeneron Pharmaceuticals, Inc. (REGN), Gilead Sciences Inc. (GILD) and Alexion Pharmaceuticals, Inc. (ALXN). While Gilead and Regeneron are Zacks Rank #1 (Strong Buy) stocks, Alexion holds a Zacks Rank #2 (Buy).